The Medical Letter on Drugs and Therapeutics
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1499
Asfotase Alfa (Strensiq) for Hypophosphatasia (online only)
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The FDA has approved asfotase alfa (Strensiq – Alexion), a recombinant form of tissue-nonspecific alkaline phosphatase, for subcutaneous treatment of perinatal-, infantile-, and juvenile-onset hypophosphatasia. Asfotase alfa is the first treatment to be approved in the US for this rare genetic metabolic disorder.

HYPOPHOSPHATASIA — Tissue-nonspecific alkaline phosphatase is essential for mineralization of bones and teeth. Hypophosphatasia is caused by mutations in the alkaline phosphatase gene. It can present at any time (in utero to adulthood) and is highly variable in severity. Perinatal- and infantile-onset hypophosphatasia can cause deformities of the chest and underdevelopment of lungs, and is often fatal. Juvenile-onset hypophosphatasia is somewhat less severe, but growth failure, skeletal deformities, and fractures can occur. Spontaneous remission seems to occur in some young adults, but ... more      

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Title: Asfotase Alfa (Strensiq) for Hypophosphatasia (online only)
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